Duchenne muscular dystrophy

Incremental clinical advances for Duchenne Muscular DystrophyJust last year, the FDA approved Exondys 51 (eteplirsen) for DMD, over the recommendations of FDA scientists and an external scientific advisory committee. Read more.
9 Newly Approved Novel Drugs
9 Newly Approved Novel DrugsIt's still early in 2017, but the FDA has been approving drugs left and right. Here's what you need to know.
Muscular dystrophy drug launch halts after pricing concernsMarathon Pharmaceuticals is temporarily pausing the launch of its drug to treat a rare form of muscular dystrophy (MD) after concerns over its $89,000 price tag.
FDA approves first drug for Duchenne muscular dystrophyEven though an FDA panel recommended against approving a new drug to treat Duchenne muscular dystrophy in April, the agency granted accelerated approval to eteplirsen injection (Exondys 51, Sarepta Therapeutics) in mid-September.
Three drug trends impacting specialty pharmacyAMCP session, “Specialty Pharmaceutical in Development,” highlighted critical trends that healthcare executives should be watching
FDA drug approvals-April 2015FDA actions in brief, priority review, breakthrough therapy designation, fast-track designations, orphan drug designations, first-time generic approvals
CEO resignation linked to FDA drug approval processSarepta Therapeutics CEO Chris Garabedian resigned earlier this week, a move that may be linked to a dispute with the Food and Drug Administration (FDA) over the approval process for its new drug, according to The Wall Street Journal.
FDA drug approvals-January 2015FDA actions in brief, recommendations for approval, fast-track designations.
Early clues to muscular dystrophy in boysA combination of 2 developmental delays in toddler boys can alert physicians to the possibility of Duchenne muscular dystrophy and lead to earlier diagnosis, a new study reports.
FDA drug approvals-July 2014FDA drug approvals, priority review, breakthrough designation, fast-track designations, orphan drug designations, first-time generic approval